Trial Condition(s):
Hemophilia A
Prophylaxis versus On-demand Therapy Through Economic Report (POTTER)
Bayer Identifier:
11856
ClinicalTrials.gov Identifier:
EudraCT Number:
Not Available
EU CT Number:
Not Available
Trial Purpose
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
The observational period will cover at least 5 years per patient.
The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.
Inclusion Criteria
- age ≥ 12 years and ≤ 55 years - severe haemophilia A (FVIII < 1%) - absence of inhibitors (Bethesda titre < 0.6 BU/ml) - Previous Treated Patients (prior exposure days > 200) - Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group - ≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group - written informed consent
Exclusion Criteria
- concomitant severe and chronic diseases or congenital skeletal malformation - unreliability of patient or likelihood of follow-up failure - presence of inhibitors or history of inhibitors (in the previous 2 years) - currently on immune tolerance treatment - hepatic cirrhosis or liver disease in rapid progression - AIDS - platelet count < 75,000/mm3 - presence of conditions that influence negatively patient´s compliance - participation in another study
Trial Summary
Enrollment Goal
Trial Dates
Phase
Could I receive a placebo?
Products
Accepts Healthy Volunteers
Where to Participate
| Locations | |
|---|---|
Locations Many Locations Many Locations, Italy | Contact Us: E-mail: [email protected] Phone: Not Available |
Trial Design
Observational study evaluating efficacy and costs of secondary prophylaxis vs on-demand therapy with Kogenate Bayer in patients with severe haemophilia A. POTTER
Trial Type:
Observational
Intervention Type:
Drug
Trial Purpose:
N/A
Allocation:
N/A
Blinding:
N/A
Assignment:
N/A
Trial Arms:
2
Primary Outcome
- Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per yearTimeframe: Every 6 months
Secondary Outcome
- Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodesTimeframe: Every 6 months
- Muscolo skeletal evaluation by Orthopedic Joint ScoreTimeframe: Every 12 months
- Radiological Evaluation by Pettersson ScoreTimeframe: Baseline and after 3-5 years
- Health related quality of lifeTimeframe: Every 12 months
- cost-effectiveness and utility, patient compliance, adverse eventsTimeframe: Every 6 months
Additional Information
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